New live biotherapeutic products will require regulatory and scientific innovation

NARBONNE, France – The Pharmabiotic Research Institute (PRI), Europe’s only regulatory expertise centre for the development of microbiome-based drug products, has announced the publication of a review titled “Live biotherapeutic products: the importance of a defined regulatory framework”. Published in the journal Experimental & Molecular Medicine, it shines a light on the need for clear scientific and pharmaceutical standards when developing and registering live biotherapeutic products (LBPs), a new and innovative type of microbiome-based drug product. Many in the general public will have heard of ‘probiotics’, which according to the WHO definition are ‘live microorganisms that when administered in adequate amounts confer a health benefit on the host…’; however the authors of this review have focused on live microorganisms as active pharmaceutical substances intended to be used in the treatment or prevention of disease in humans. It is an in-depth treatment of the latest regulatory innovations and ‘quality-by-design’ development principles which will be critical in the appropriate assessment of these biologic medicines’ quality, efficacy and safety. This work draws on years of interactions with leading biotechs in the field, discussions with national and European health authorities (mainly through scientific advice), as well as extensive research of the existing European and other regions’ relevant pharmaceutical frameworks. The PRI is a European non-profit organization, and this topic is a key example of the type of regulatory challenges that its members are striving to overcome in their pursuit of microbiome medicinal product development and registration for the EU Market.

“Ideally, this article will help stakeholders in the field of microbiome-based products understand that the active substance or the ingredient of a product does not define its regulatory status. Thus, ‘probiotic’ substances can be developed as dietary supplements, food ingredients or drugs, depending on what these substances are intended to do and to whom,” says co-author Dr. Alice Rouanet (PRI).

Dr. Magali Cordaillat-Simmons, Executive Director of the PRI continues, “In a field where regulatory confusion often exists we perceived a strong need for clarification of the general concepts and specific constraints associated with ‘probiotic strains’ intended for prevention or treatment of a human disease, i.e. in the context of their registration as biological drug products.”

Similar to all products intended to prevent or treat diseases, LBPs will have to be registered as medicinal products to reach market in the US and in Europe. LBPs under the current European legislative framework have no ‘separate status’, and “…are by nature considered biological medicinal products as the active substances are live microorganisms, which are biological substances…LBPs have to comply with the biological medicinal product legislative and regulatory framework.” The authors address several aspects of pharmaceutical development principles, notably; ‘How can the quality of LBPs be ensured and demonstrated?’, ‘How can the safety of LBPs be demonstrated?’, and ‘How can the efficacy of LBPs be demonstrated?’.

Pr. Bruno Pot, Science Director for Yakult Europe and PRI President concludes, “New medicinal products directed to the gut microbiota pose big scientific challenges, because of the complexity and variability of the microbiota. The regulatory challenge, however, is also real, maybe because of the complexity and variation of the possible paths to success! Time and money can be gained in making decisions as early as possible on the regulatory path to follow: it will help to decide on the study planning and result in the preparation of a valid and credible dossier for the evaluation by competent authorities. Our hope is that this paper could assist you somehow in this complex process.”

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Review article published in Experimental & Molecular Biology: Live biotherapeutic products: the importance of a defined regulatory framework

The Pharmabiotic Research Institute (PRI) is a neutral and financially independent non-profit association (according to French law: Association loi 1901), which believes that the microbiome is the future of human medicine. The PRI applies a unique collaborative approach to improve market access and provide technical and regulatory intelligence for its members in the microbiome & human health space. Understanding the microbiome and its role in human health, however, represents a paradigm shift in how medicines will be developed, as innovation and industry often outpace regulation. The PRI was founded to make Microbiotic Medicinal Products (MMPs) a therapeutic reality in Europe. Our mission is to facilitate the conversation between European regulators and MMP developers and their partners. By being the main representative organization interrogated by the competent authorities regarding the future regulatory framework for MMPs, the PRI has become the first network for the European microbiome pharmaceutical supply chain, federating fundamental actors from the industry, such as stakeholders involved in the development, investment, production and commercialization of MMPs.